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ORIGINAL RESEARCH article

Front. Public Health

Sec. Health Economics

Volume 13 - 2025 | doi: 10.3389/fpubh.2025.1597550

This article is part of the Research TopicImmunization Economics: Balancing Costs, Outcomes, and Equity in Public HealthView all articles

Multinational cost-effectiveness analysis of pembrolizumab combined with chemotherapy as first-line treatment for advanced biliary tract cancer

Provisionally accepted
Yuxuan  XieYuxuan XieQuanyi  LiuQuanyi LiuShanrui  XiaoShanrui XiaoXia  LiXia LiLin  QiuLin QiuYun  GuYun Gu*
  • The First Affiliated Hospital of Dali University, Dali, China

The final, formatted version of the article will be published soon.

Background: Immunotherapy is a promising treatment for advanced biliary tract cancer. However, the cost-effectiveness of pembrolizumab combined with gemcitabine and cisplatin remains unclear across different healthcare systems. This study evaluates its international cost-effectiveness in four countries.A partitioned survival analysis model was developed using data from the KEYNOTE-966 trial to compare costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) of pembrolizumab plus gemcitabine and cisplatin versus gemcitabine and cisplatin alone, from the healthcare system perspective of China, Japan, the United States, and Switzerland. Sensitivity and scenario analyses were used to identify key factors influencing the ICER.The base-case analysis showed that pembrolizumab plus gemcitabine and cisplatin provided additional quality-adjusted life year gains of 0.14 in China, Japan, and the United States, and 0.15 in Switzerland. The incremental cost-effectiveness ratios in all four countries exceeded their respective willingness-to-pay thresholds, indicating limited cost-effectiveness. Sensitivity analysis identified drug price and utility value of progression-free survival as key factors. In the United States, Japan, and Switzerland, an 80-95% reduction in pembrolizumab's price was necessary for cost-effectiveness, while in China, a reduction greater than 95% was required.Conclusions: Despite clinical benefits, pembrolizumab combined with chemotherapy for advanced biliary tract cancer is not cost-effective at current prices and willingness-to-pay thresholds. Adjusting drug pricing and healthcare policies is crucial for enhancing the global economic viability of this treatment strategy.

Keywords: Biliary tract cancer, Pembrolizumab, immune checkpoint inhibitors, multinational economic evaluation, cost-effectiveness analysis

Received: 21 Mar 2025; Accepted: 22 Jul 2025.

Copyright: © 2025 Xie, Liu, Xiao, Li, Qiu and Gu. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Yun Gu, The First Affiliated Hospital of Dali University, Dali, China

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